Originally Aired: September 20, 2023
Time: 9:00 am PT, 12:00 pm ET, 18:00 CET


Using antisense oligonucleotides (ASOs) to treat patients with ultra-rare diseases is an incredibly exciting area. But the road from drug design to treatment is a long (and expensive) one.

On this GEN Live, we’ll discuss where ASO research is now, the challenges it faces as it moves forward, and the innovative new ways that those challenges are being overcome to treat patients. Dr. Stan Crooke, a pioneer in the field, founder of Ionis Pharmaceuticals and the n-Lorem Foundation and Dr. Punit Seth, senior vice president at Alnylam Pharmaceuticals will join us for a broad discussion on the topic.

GEN moderator:
• Julianna LeMieux, PhD – Deputy Editor-in-Chief, GEN 


A live Q&A session followed the presentation, offering a chance to pose questions to our expert panelist.

Stan Crooke
Stan Crooke, MD, PhD
Founder and CEO, n-Lorem Foundation
Founder and Former CEO, Ionis Pharmaceuticals
Punit Seth
Punit Seth, PhD
SVP Research
Alnylam Pharmaceuticals